Ignite Creation Date:
2025-12-24 @ 10:37 PM
Ignite Modification Date:
2025-12-25 @ 8:09 PM
Study NCT ID:
NCT01923935
Status:
UNKNOWN
Last Update Posted:
2013-08-19
First Post:
2013-08-09
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
Busulfan and Melphalan Conditioning in Multiple Myeloma
Sponsor:
Chonnam National University Hospital
Organization:
Study Overview
Official Title:
A Phase 2, Open-label, Prospective, Multicenter Study to Evaluate the Efficacy of Intravenous Busulfan and Melphalan as a Conditioning Regimen in Patients With Multiple Myeloma Undergoing Autologous Stem Cell Transplantation
Status:
UNKNOWN
Status Verified Date:
2013-08
Last Known Status:
RECRUITING
Delayed Posting:
No
If Stopped, Why?:
Not Stopped
Has Expanded Access:
False
If Expanded Access, NCT#:
N/A
Has Expanded Access, NCT# Status:
N/A
Acronym:
None
Brief Summary:
The purpose of this study is to determine whether intravenous busulfan and melphalan as a conditioning regimen is effective in the treatment of multiple myeloma undergoing autologous stem cell transplantation.
Detailed Description:
Title
* A phase 2, open-label, prospective, multicenter study to evaluate the efficacy of intravenous busulfan and melphalan as a conditioning regimen in patients with multiple myeloma (MM) undergoing autologous stem cell transplantation (ASCT)
Principal Investigator
* Je-Jung Lee (Chonnam National University Hwasun Hospital)
Co-investigators
* Hyeon Seok Eom (National Cancer Center)
* Kihyun Kim (Samsung Medical Center)
* Chang Ki Min (Seoul St. Mary's Hospital)
* Soo Jung Kim (Severance Hospital)
* Sung Soo Yoon (Seoul National University Hospital)
* Jae Hoon Lee (Gachon University Gil Hospital)
* Yeung-Chul Mun (Ewha Womans University Mokdong Hospital)
Duration
* 2 years
Study phase
* Phase II
Patients
* Patients with multiple myeloma who undergo autologous stem cell transplantation
Objectives(end points)
* Primary objective:
1. Treatment response up to 2 months after ASCT
2. Safety and toxicity (frequency of grade 3 or worse toxicities) of the conditioning regimen
* Secondary objective:
1. Progression free survival (PFS)
2. Overall survival (OS)
Total patients
* Initial 105 evaluable patients
* Complete Response (CR) rate of 200mg/m2 melphalan conditioning chemotherapy followed by ASCT in patients with newly diagnosed MM was about 26% and CR rate of busulfan and melphalan conditioning chemotherapy followed by ASCT in patients with newly diagnosed MM was about 40%. If the CR rate of busulfan and melphalan conditioning chemotherapy followed by ASCT is more than 40%, this combination will be accepted as active conditioning regimen that may be worth for investigating in phase III trial. But, if the CR rate of this regimen is lower than 26%, this has not a merit than 200mg/m2 melphalan conditioning chemotherapy. Based upon the above assumption, this trial was designed by using Simon's optimal two-stage testing procedure. Assuming a target level of interest, p1=0.4, and a lower activity level, p0=0.26 and drop rate 0.1. Initially 44 patients will be accrued. If 13 or more CR rate were observed, the trial will be continued. Accrual will be planned to a total of 105 patients, If total 35 or more patients were assessed as CR, busulfan and melphalan conditioning regimen will be accepted as active regimen, This design provides probability ≤ 0.05 of accepting drugs worse than p0 and probability ≤ 0.20 of rejecting drugs better than p1.if we assume that drop-out rate is 10%, total accrual patient will be 105
Treatment Schedule
* Busulfan 3.2 mg/kg/day iv once daily over 3 hours(day-6 \~ day-4)
* Melphalan 70 mg/m2/day iv once daily over 30 minutes(day-3 \~ day-1). If Creatinine Clearance (mL/min) \< 50, reduce to 50 mg/m2. If Creatinine Clearance (mL/min) \< 30, excluded from the this trial
Informed consent
* Written informed consent must be obtained before any study-specific screening procedures are performed
Screening
* Baseline assessments should be made within 28 days before treatment start:
1. Demographic data (date of birth and sex)
2. Eastern Cooperative Oncology Group performance status
3. Vital signs and physical examination (including height, and weight)
4. Medical history (including previous diseases/treatments and concomitant diseases/ treatments)
5. Hematology - complete blood counts with differential count examination
6. Serum electrolytes (Na, K, Cl, Ca, phosphorus)
7. Serum lactate dehydrogenase
8. Hepatitis B virus/hepatitis C virus/HIV serology (If serologic tests were conducted within 6 months prior to screening, retests are not required)
9. Serum Beta2-microglobulin
10. Quantitative serum M-protein (Serum protein electrophoresis), including immunofixation or immune electrophoresis
11. Quantitative urine M-protein in 24 hrs urine (Urine protein electrophoresis), including immunofixation or immune electrophoresis
12. Serum free light chain assay
13. Creatinine clearance if increased serum creatinine
14. ECG
15. multigated radionuclide angiography or 2D ECHO
16. Chest X-ray, Radiographic skeletal survey including skull, pelvis, vertebral column and long bones
17. Bone marrow aspiration and biopsy with chromosome study, and flow cytometry or immunohistochemistry
Assessment
* Primary outcome measure
1. To evaluate the objective responses after ASCT, the guidelines from the International Myeloma Working (IMW) Group uniform response criteria will be used
2. Response Criteria for Multiple Myeloma the guidelines from the IMW Group uniform response criteria + Add for criteria of near CR (Immunofixation positive CR)
3. Serum free light chain study will be added at the every evaluation of response
4. To confirm the stringent complete response (sCR) after ASCT, flow cytometry or immunohistochemistry will be used
5. NCI Common Toxicity Criteria for Adverse Effects version 4.0 will be used for the examination of toxicities
* Secondary outcome measure
1. PFS will be defined from the time of ASCT to the time of first sign of disease progression or death
2. OS will be defined as the period from the time of ASCT to the date of the last follow-up or death from any cause
* A phase 2, open-label, prospective, multicenter study to evaluate the efficacy of intravenous busulfan and melphalan as a conditioning regimen in patients with multiple myeloma (MM) undergoing autologous stem cell transplantation (ASCT)
Principal Investigator
* Je-Jung Lee (Chonnam National University Hwasun Hospital)
Co-investigators
* Hyeon Seok Eom (National Cancer Center)
* Kihyun Kim (Samsung Medical Center)
* Chang Ki Min (Seoul St. Mary's Hospital)
* Soo Jung Kim (Severance Hospital)
* Sung Soo Yoon (Seoul National University Hospital)
* Jae Hoon Lee (Gachon University Gil Hospital)
* Yeung-Chul Mun (Ewha Womans University Mokdong Hospital)
Duration
* 2 years
Study phase
* Phase II
Patients
* Patients with multiple myeloma who undergo autologous stem cell transplantation
Objectives(end points)
* Primary objective:
1. Treatment response up to 2 months after ASCT
2. Safety and toxicity (frequency of grade 3 or worse toxicities) of the conditioning regimen
* Secondary objective:
1. Progression free survival (PFS)
2. Overall survival (OS)
Total patients
* Initial 105 evaluable patients
* Complete Response (CR) rate of 200mg/m2 melphalan conditioning chemotherapy followed by ASCT in patients with newly diagnosed MM was about 26% and CR rate of busulfan and melphalan conditioning chemotherapy followed by ASCT in patients with newly diagnosed MM was about 40%. If the CR rate of busulfan and melphalan conditioning chemotherapy followed by ASCT is more than 40%, this combination will be accepted as active conditioning regimen that may be worth for investigating in phase III trial. But, if the CR rate of this regimen is lower than 26%, this has not a merit than 200mg/m2 melphalan conditioning chemotherapy. Based upon the above assumption, this trial was designed by using Simon's optimal two-stage testing procedure. Assuming a target level of interest, p1=0.4, and a lower activity level, p0=0.26 and drop rate 0.1. Initially 44 patients will be accrued. If 13 or more CR rate were observed, the trial will be continued. Accrual will be planned to a total of 105 patients, If total 35 or more patients were assessed as CR, busulfan and melphalan conditioning regimen will be accepted as active regimen, This design provides probability ≤ 0.05 of accepting drugs worse than p0 and probability ≤ 0.20 of rejecting drugs better than p1.if we assume that drop-out rate is 10%, total accrual patient will be 105
Treatment Schedule
* Busulfan 3.2 mg/kg/day iv once daily over 3 hours(day-6 \~ day-4)
* Melphalan 70 mg/m2/day iv once daily over 30 minutes(day-3 \~ day-1). If Creatinine Clearance (mL/min) \< 50, reduce to 50 mg/m2. If Creatinine Clearance (mL/min) \< 30, excluded from the this trial
Informed consent
* Written informed consent must be obtained before any study-specific screening procedures are performed
Screening
* Baseline assessments should be made within 28 days before treatment start:
1. Demographic data (date of birth and sex)
2. Eastern Cooperative Oncology Group performance status
3. Vital signs and physical examination (including height, and weight)
4. Medical history (including previous diseases/treatments and concomitant diseases/ treatments)
5. Hematology - complete blood counts with differential count examination
6. Serum electrolytes (Na, K, Cl, Ca, phosphorus)
7. Serum lactate dehydrogenase
8. Hepatitis B virus/hepatitis C virus/HIV serology (If serologic tests were conducted within 6 months prior to screening, retests are not required)
9. Serum Beta2-microglobulin
10. Quantitative serum M-protein (Serum protein electrophoresis), including immunofixation or immune electrophoresis
11. Quantitative urine M-protein in 24 hrs urine (Urine protein electrophoresis), including immunofixation or immune electrophoresis
12. Serum free light chain assay
13. Creatinine clearance if increased serum creatinine
14. ECG
15. multigated radionuclide angiography or 2D ECHO
16. Chest X-ray, Radiographic skeletal survey including skull, pelvis, vertebral column and long bones
17. Bone marrow aspiration and biopsy with chromosome study, and flow cytometry or immunohistochemistry
Assessment
* Primary outcome measure
1. To evaluate the objective responses after ASCT, the guidelines from the International Myeloma Working (IMW) Group uniform response criteria will be used
2. Response Criteria for Multiple Myeloma the guidelines from the IMW Group uniform response criteria + Add for criteria of near CR (Immunofixation positive CR)
3. Serum free light chain study will be added at the every evaluation of response
4. To confirm the stringent complete response (sCR) after ASCT, flow cytometry or immunohistochemistry will be used
5. NCI Common Toxicity Criteria for Adverse Effects version 4.0 will be used for the examination of toxicities
* Secondary outcome measure
1. PFS will be defined from the time of ASCT to the time of first sign of disease progression or death
2. OS will be defined as the period from the time of ASCT to the date of the last follow-up or death from any cause
Study Oversight
Has Oversight DMC:
True
Is a FDA Regulated Drug?:
None
Is a FDA Regulated Device?:
None
Is an Unapproved Device?:
None
Is a PPSD?:
None
Is a US Export?:
None
Is an FDA AA801 Violation?: